Gene-edited stem cells: a lasting fix for sickle cell?
NCT ID NCT06155500
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study follows 4 people with sickle cell disease who previously received a one-time treatment of their own gene-edited stem cells (OTQ923). Researchers are checking for delayed side effects, such as new cancers or blood disorders, and measuring how long fetal hemoglobin production lasts. The goal is to see if the treatment remains safe and effective over time.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
OTQ923 (gene-edited stem cells)
What this could lead to
If successful, this could show that a single treatment with gene-edited stem cells provides lasting control of sickle cell disease symptoms.
What could go wrong
This is a very small, early-phase follow-up study with only 4 participants, so results may not apply broadly. Long-term risks like new cancers or blood disorders are still being checked.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Memorial Sloan Kettering Cancer Ctr
New York, New York, 10065, United States
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St Jude Childrens Research Hospital
Memphis, Tennessee, 38105, United States
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University of Chicago
Chicago, Illinois, 60637, United States